Texas Children's and Baylor Awarded Up to $17.25 Million to Join ARIA IMPACT Network for Autism Clinical Trials
核心洞察
Dr. Jimmy Holder and his team at Texas Children's Hospital and Baylor College of Medicine have received up to $17.25 million from ARIA to join the IMPACT Network.
The IMPACT Network comprises 12 collaborative sites worldwide aimed at accelerating clinical trial readiness for autism therapies, including rare genetic and non-genetic forms.
The team will contribute to a short-term natural history and clinical endpoint study focused on children with profound autism.
A major new grant is poised to accelerate the translation of autism research into clinical therapies. Dr. Jimmy Holder, associate professor in pediatrics at Baylor College of Medicine and principal investigator at the Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital, and his team have been awarded up to $17.25 million from Aligning Research to Impact Autism (ARIA) (搜索) to become part of the Innovative Medicine and Precision Approaches to Clinical Trials (IMPACT) Network.
As part of the IMPACT Network, Holder and his team will serve as one of an initial 12 collaborative sites around the world designed to accelerate clinical trial readiness and implementation. The network aims to find promising therapies for different forms of autism, including both those related to a rare genetic mutation and those without an identified genetic cause.
"The ARIA initiative's support could not come at a better time, as our understanding of autism has advanced so that we now stand at an exciting precipice, ready to translate that progress into clinical trials and life-changing treatments for patients and families," said Holder. "Joining the ARIA IMPACT Network will allow Baylor and Texas Children's Hospital to accelerate our breakthrough work to bring effective therapies to people with autism and related neurodevelopmental disorders."
A Foundation Built on Years of Autism Research
The selection of the Duncan Neurological Research Institute at Texas Children's and Baylor reflects the organizations' many years of investment in autism research and clinical care. Since the institute's creation in 2010, Duncan NRI researchers have discovered or co-discovered the genetic basis of numerous neurological and developmental disorders, including Rett syndrome (搜索), an autism spectrum disorder (搜索).
An international leader in genetics, neuroscience, and pediatrics with an emphasis on multidisciplinary collaboration, the Duncan NRI is well-positioned to contribute to the ARIA initiative's scientific discovery. As part of this network, Baylor and Texas Children's will contribute to a short-term natural history and clinical endpoint study, which aims to better understand how autism and related neurodevelopmental conditions develop and change over time in children with profound autism.
Focus on SHANK3 (搜索) and SYNGAP1 Genes
Holder's own research lab focuses on the SHANK3 (搜索) and SYNGAP1 genes, which cause severe neurodevelopmental disorders, such as autism spectrum disorders and intellectual disabilities, when mutations occur. By identifying these mutations and the processes they affect, his team is working to find therapeutic targets for these disorders.
ARIA is an initiative that connects emerging research, insights, and promising technologies from across scientific fields with the goal of advancing scientific discovery and creating more therapeutic opportunities for people with profound autism and people on the spectrum.
"The ARIA IMPACT Network was built on the premise that no single institution can generate the scale and quality of evidence this field needs on its own," said Ekemini Riley, PhD, managing director of ARIA. "The team at Texas Children's Hospital brings the expertise and dedication that make this kind of coordinated science possible, and we are glad to have them as partners in advancing therapeutic opportunities for autistic people and related neurodevelopmental conditions."
Multidisciplinary Collaboration
The project brings together a broad team of collaborators from Baylor College of Medicine and Texas Children's Hospital, including Dr. Holly Harris, associate professor of pediatrics and developmental-behavioral pediatrics specialist; Dr. Mirjana Maletic-Savatic, professor of pediatrics–neurology and Duncan NRI investigator; Dr. Michael Wangler, associate professor of molecular and human genetics and Duncan NRI investigator; Dr. Davut Pehlivan, assistant professor of pediatrics–neurology; Dr. Daniel Curry, professor of neurosurgery and director of functional neurosurgery and epilepsy surgery at Texas Children's; Dr. Robin Kochel, associate professor of pediatrics–psychology and associate director for research at the Autism Center at Texas Children's; Dr. Bernhard Suter, associate professor of pediatrics–neurology; Dr. Anne Anderson, professor of pediatrics–neurology and chief of clinical neurophysiology at Texas Children's; Dr. Matthew McGinley, associate professor of neuroscience; Dr. Zhandong Liu, associate professor of pediatrics–neurology and chief of computational sciences at Texas Children's; and Dr. Leandra Berry, assistant professor of pediatrics and pediatric neuropsychologist at Texas Children's.